title: Preapproval and postapproval availability of published comparative efficacy research on biological agents.
abstract: OBJECTIVE
  Preapproval and postapproval availability of published comparative efficacy studies on biological agents approved between 2000 and 2010 was investigated.
  
  
  METHODS
  Approval packages published on the Food and Drug Administration (FDA) website were examined for all biological agents approved between 2000 and 2010 to determine if comparative efficacy studies were available at the time of FDA approval. The availability of comparative efficacy studies published subsequent to approval was determined by searching PubMed for randomized, active-controlled experimental or observational study designs that measured efficacy as the primary endpoint and were relevant to the original FDA-approved indication.
  
  
  RESULTS
  From 2000 to 2010, 107 biological agents were approved by FDA. Of the biological agents with alternative treatments, 54.6% had comparative efficacy data available at the time of approval. Although standard-reviewed biological agents were more likely to have comparative efficacy trials included in the FDA approval packages than priority-reviewed biological agents, statistically significant differences are unlikely. Subsequent to approval, 58.1% of biological agents had at least one published comparative efficacy trial, representing a 3.5% absolute increase in the availability of comparative efficacy studies since the time of approval. Vaccines and biological agents in the hematologic diseases, oncology, and miscellaneous diseases classes had fewer published postapproval comparative efficacy studies per agent compared with the overall group of biological agents.
  
  
  CONCLUSIONS
  Nearly half of all biological agents approved for marketing between 2000 and 2010 lacked publicly accessible, active-controlled efficacy studies at the time of drug approval; a slightly greater proportion of biological agents had comparative efficacy data published subsequent to their approval.
authors.affiliation: McWhorter School of Pharmacy, Samford University, Birmingham, AL 35229, USA. rahutchi@samford.edu
authors.forename: Rachel Hutchins
authors.initials: RH
authors.lastname: Thomas
authors.forename: Maisha Kelly
authors.initials: MK
authors.lastname: Freeman
authors.forename: Peter J
authors.initials: PJ
authors.lastname: Hughes
mesh_terms: D001688:Biological Products / Q000627:therapeutic use*; D016208:Databases, Factual / Q000592:standards; D017277:Drug Approval / Q000379:methods; D006801:Humans; D016032:Randomized Controlled Trials as Topic / Q000592:standards; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration / Q000592:standards*
chemical_list: D001688:Biological Products